Department of Neurology, Medical University of Vienna, Waehringer Guertel 18-20, 1090, Vienna, Austria.
Department of Neurology, Karl-Franzens University, Graz, Austria.
J Neurol. 2023 Aug;270(8):3779-3786. doi: 10.1007/s00415-023-11686-2. Epub 2023 Apr 19.
With the approval of natalizumab in Europe in 2006, the Austrian Multiple Sclerosis Therapy Registry (AMSTR) was established. Here, we present data from this registry about effectiveness and safety of natalizumab in patients treated up to 14 years.
PATIENTS/METHODS: Data retrieved from the AMSTR contained baseline characteristics and biannual documentation of annualised relapse rate (ARR) and Expanded Disability Status Scale (EDSS) score as well as adverse events and reasons for discontinuation on follow-up visits.
A total of 1596 natalizumab patients (71% women, n = 1133) were included in the analysis and the observed treatment duration ranged from 0 to 164 months (13.6 years). The mean ARR was 2.0 (SD = 1.13) at baseline, decreasing to 0.16 after 1 year and 0.01 after 10 years. A total of 325 patients (21.6%) converted to secondary progressive multiple sclerosis (SPMS) during the observational period. Of 1502 patients, 1297 (86.4%) reported no adverse events (AE) during follow-up visits. The most common reported AEs were infections and infusion-related reactions. John Cunningham virus (JCV) seropositivity was the most common specified reason for treatment discontinuation (53.7%, n = 607). There were five confirmed cases of Progressive Multifocal Leukoencephalopathy (PML) with 1 death.
The effectiveness of natalizumab in patients with active relapsing-remitting multiple sclerosis (RRMS) could be confirmed in our real-world cohort even after follow-up of up to 14 years, though after year 10, there were less than 100 remaining patients. A low number of AE were reported in this nationwide registry study, establishing Natalizumab's favourable safety profile during long-term use.
随着 2006 年那他珠单抗在欧洲获得批准，奥地利多发性硬化症治疗登记处（AMSTR）成立。在这里，我们展示了该登记处关于那他珠单抗治疗患者的有效性和安全性的数据，随访时间长达 14 年。
患者/方法：从 AMSTR 中检索到的数据包含基线特征和每两年一次的年复发率（ARR）和扩展残疾状态量表（EDSS）评分的记录，以及不良事件和随访时停药的原因。
共有 1596 名那他珠单抗患者（71%为女性，n=1133）纳入分析，观察到的治疗持续时间从 0 到 164 个月（13.6 年）不等。基线时平均 ARR 为 2.0（SD=1.13），治疗 1 年后降至 0.16，治疗 10 年后降至 0.01。在观察期间，共有 325 名患者（21.6%）转为继发进展性多发性硬化症（SPMS）。在 1502 名患者中，1297 名（86.4%）在随访期间未报告不良事件（AE）。报告的最常见的 AE 是感染和输注相关反应。约翰·坎宁安病毒（JCV）血清阳性是最常见的指定停药原因（53.7%，n=607）。有 5 例确诊的进行性多灶性白质脑病（PML），其中 1 例死亡。
即使在长达 14 年的随访后，我们的真实世界队列仍证实了那他珠单抗在活动性复发缓解型多发性硬化症（RRMS）患者中的有效性，尽管在第 10 年后，仍有不到 100 名患者存活。这项全国性登记研究报告的不良事件数量较少，证实了那他珠单抗在长期使用中的良好安全性。
